US2014187595A1PendingUtilityA1

Methods and Compositions Comprising AMPK Activator (Metformin/Troglitazone) for the Treatment of Myotonic Dystrophy Type 1 (DM1)

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Assignee: INST NAT SANTE RECH MEDPriority: Apr 2, 2010Filed: Jan 10, 2014Published: Jul 3, 2014
Est. expiryApr 2, 2030(~3.7 yrs left)· nominal 20-yr term from priority
Inventors:Sandrine BaghdoyanMarc PeschanskiDelphine LaustriatJacqueline Gide
A61P 43/00A61P 21/00A61K 31/426G01N 33/68A61K 31/155A61K 31/4436A61K 31/427
48
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Claims

Abstract

The present invention relates to methods and compositions for the treatment of Myotonic Dystrophy type 1 (DM1) with an AMPK activator <eq.metformin or troglizazone>.

Claims

exact text as granted — not AI-modified
1 .- 14 . (canceled) 
     
     
         15 . A method for treating and/or preventing Myotonic Dystrophy type 1 (DM1) in a presymptomatic DM1 patient, comprising administering an effective amount of an AMPK activator to the presymptomatic DM1 patient. 
     
     
         16 . The method according to  claim 15 , wherein said patient is not suffering from insulin resistance. 
     
     
         17 . The method according to  claim 15 , wherein said patient is not suffering from diabetes. 
     
     
         18 . The method according to  claim 15 , wherein said AMPK activator is selected from the group consisting of Metformin, Thiazolidinediones, Adiponectin, Leptin, Ciliary Neurotrophic Factor (CNTF), Ghrelin/cCannabinoids, Interleukin-6, alpha-Lipoic Acid alkaloids, bitter melon extracts, resveratrol, epigallocathechin gallate, berberine, quercetin, ginsenoside, curcumin, caffeic acid phenethyl ester, theaflavin, A-769662, PT1, Thienopyridone derivatives, imidazole derivatives, and thiazole derivatives. 
     
     
         19 . The method according to  claim 15 , wherein said AMPK activator is metformin or a thiazolidinedione. 
     
     
         20 . The method according to  claim 19 , wherein said AMPK activator is metformin. 
     
     
         21 . The method according to  claim 19 , wherein said thiazolidinedione is selected from the group consisting of troglitazone, rosiglitazone, and pioglitazone. 
     
     
         22 . The method according to  claim 15 , comprising administering at least a first and a second AMPK activator to said patient, wherein the first AMPK activator is metformin and the second AMPK activator is a thiazolidinedione selected from the group consisting of troglitazone, rosiglitazone, and pioglitazone. 
     
     
         23 . A method for correcting splicing defects in a myotonic dystrophy type 1 (DM1) cell, comprising contacting the DM1 cell with an effective amount of an AMPK activator. 
     
     
         24 . The method according to  claim 23 , wherein said AMPK activator is selected from the group consisting of Metformin, Thiazolidinediones, Adiponectin, Leptin, Ciliary Neurotrophic Factor (CNTF), Ghrelin/cCannabinoids, Interleukin-6, alpha-Lipoic Acid alkaloids, bitter melon extracts, resveratrol, epigallocathechin gallate, berberine, quercetin, ginsenoside, curcumin, caffeic acid phenethyl ester, theaflavin, A-769662, PT1, Thienopyridone derivatives, imidazole derivatives, and thiazole derivatives. 
     
     
         25 . The method according to  claim 23 , wherein said AMPK activator is metformin or a thiazolidinedione. 
     
     
         26 . The method according to  claim 25 , wherein said AMPK activator is metformin. 
     
     
         27 . The method according to  claim 25 , wherein said thiazolidinedione is selected from the group consisting of troglitazone, rosiglitazone or pioglitazone. 
     
     
         28 . A method for modulating the ratio of the levels of expression of insulin receptor isoform, INSR-A to insulin receptor isoform, INSR-B in a cell, comprising contacting the cell with an effective amount of an AMPK activator. 
     
     
         29 . The method of  claim 28 , wherein the amount of AMPK activator is effective to decrease the amount of ELAVL1 protein in the cytoplasm of the cell, relative to the amount of ELAVL1 protein in the cytoplasm of the cell in the absence of the AMPK1 activator. 
     
     
         30 . The method of  claim 28 , wherein the amount of AMPK activator is effective to increase the import of ELAVL1 protein to the nucleus of the cell, relative to the import of ELAVL1 protein to the nucleus of the cell in the absence of the AMPK1 activator. 
     
     
         31 . The method according to  claim 28 , wherein said AMPK activator is selected from the group consisting of Metformin, Thiazolidinediones, Adiponectin, Leptin, Ciliary Neurotrophic Factor (CNTF), Ghrelin/cCannabinoids, Interleukin-6, alpha-Lipoic Acid alkaloids, bitter melon extracts, resveratrol, epigallocathechin gallate, berberine, quercetin, ginsenoside, curcumin, caffeic acid phenethyl ester, theaflavin, A-769662, PT1, Thienopyridone derivatives, imidazole derivatives, and thiazole derivatives. 
     
     
         32 . The method according to  claim 28 , wherein said AMPK activator is metformin or a thiazolidinedione. 
     
     
         33 . The method according to  claim 32 , wherein said AMPK activator is metformin. 
     
     
         34 . The method according to  claim 32 , wherein said thiazolidinedione is selected from the group consisting of troglitazone, rosiglitazone, and pioglitazone.

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